• U.S. FDA grants Priority Review to dara­tu­mu­mab
• PDUFA target date has been set to March 9, 2016

Copenhagen (Press Release) – Genmab A/S (OMX: GEN) announced today that the U.S. Food and Drug Admin­istra­tion (FDA) has granted Priority Review to the Biologics License Application (BLA) for dara­tu­mu­mab. The BLA is for dara­tu­mu­mab as a treat­ment for patients with multiple myeloma who have received at least three dif­fer­en­t lines of ther­apy in­­clud­ing both a pro­te­a­some inhibitor and an immuno­modu­la­tory agent (IMiD) or who are double refractory to a pro­te­a­some inhibitor and an IMiD. A rolling BLA sub­mission was started by Genmab’s licensing partner, Janssen Biotech, Inc. in June and was com­pleted on July 9, 2015. In August 2012, Genmab granted Janssen an exclusive world­wide license to develop, manu­fac­ture and com­mer­cial­ize dara­tu­mu­mab.

Priority Review is an FDA desig­na­tion for drugs that treat a serious con­di­tion and may provide a sig­nif­i­cant im­prove­ment in safety or efficacy. The FDA aims to com­plete its review of the dara­tu­mu­mab BLA within six months and has assigned a Prescription Drug User Fee Act (PDUFA) target date of March 9, 2016.

“We are pleased that the FDA has granted Priority Review for dara­tu­mu­mab in double refractory multiple myeloma. If approved, dara­tu­mu­mab has the poten­tial to make a real dif­fer­ence in the lives of people who have run out of other treat­ment options for multiple myeloma,” said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The BLA sub­mission in­cludes data from the Phase II study (Sirius MMY2002) of dara­tu­mu­mab in multiple myeloma patients who have received at least three prior lines of ther­apy in­­clud­ing both a PI and an IMiD, or who are double refractory to a PI and an IMiD. However, safety and efficacy data from the Phase I/II study (GEN501) and safety data from three other studies, have also been in­cluded in the BLA sub­mission. Dara­tu­mu­mab received a Break­through Therapy Desig­na­tion for this indi­ca­tion from the FDA in May 2013.

About multiple myeloma

Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is char­ac­ter­ized by an excess proliferation of plasma cells.¹ Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lym­phoma.² Approximately 26,850 new patients will be diag­nosed with multiple myeloma and approx­i­mately 11,240 people will die from the disease in the U.S. in 2015.³ Globally, it is esti­mated that 124,225 people will be diag­nosed and 87,084 will die from the disease in 2015.⁴ While some patients with multiple myeloma have no symp­toms at all, most patients are diag­nosed due to symp­toms which can in­clude bone problems, low blood counts, cal­cium elevation, kidney problems or in­fec­tions.⁵ Patients who relapse after treat­ment with standard ther­a­pies, in­­clud­ing PIs or IMiDs, have poor prognoses and few treat­ment op­tions.⁶

About dara­tu­mu­mab

Daratumumab is an inves­ti­ga­tional human IgG1k mono­clonal anti­body (mAb) that binds with high affinity to the CD38 molecule, which is highly ex­pressed on the surface of multiple myeloma cells. It induces rapid tumor cell death through multiple immune-mediated mech­a­nisms⁷, in­­clud­ing complement-dependent cyto­toxicity7, anti­body-dependent cellular phago­cytosis⁸ and anti­body-dependent cellular cyto­toxicity⁷, as well as via induction of apop­tosis⁹. Five Phase III clin­i­cal studies with dara­tu­mu­mab in re­lapsed and front­line settings are cur­rently ongoing. Additional studies are ongoing or planned to assess its poten­tial in other malignant and pre-malig­nant diseases on which CD38 is ex­pressed, such as smol­der­ing myeloma and non-Hodgkin lym­phoma. Dara­tu­mu­mab has been granted Break­through Therapy Desig­na­tion from the US FDA.

About Genmab

Genmab is a publicly traded, inter­na­tional bio­technology com­pany specializing in the creation and devel­op­ment of dif­fer­en­ti­ated anti­body thera­peutics for the treat­ment of cancer. Founded in 1999, the com­pany cur­rently has one mar­keted anti­body, Arzerra® (ofatumumab) for the treat­ment of certain chronic lym­pho­cytic leukemia indi­ca­tions and dara­tu­mu­mab in clin­i­cal devel­op­ment for multiple myeloma and non-Hodgkin’s lym­phoma, in addi­tion to other clin­i­cal pro­grams, and an inno­va­tive pre-clinical pipe­line. Genmab's tech­nol­o­gy base consists of val­i­dated and pro­pri­e­tary next generation anti­body tech­nolo­gies - the DuoBody® plat­form for generation of bispecific anti­bodies, and the HexaBody® plat­form which creates effector function en­hanced anti­bodies. Genmab's deep anti­body expertise is ex­pec­ted to provide a stream of future prod­uct can­di­dates. Partnering of selected inno­va­tive prod­uct can­di­dates and tech­nolo­gies is a key focus of Genmab’s strategy and the com­pany has alliances with top tier pharma­ceu­tical and bio­technology com­pa­nies. For more in­­for­ma­tion visit www.genmab.com.

This Company Announcement con­tains for­ward looking state­ments. The words “believe”, “expect”, “antic­i­pate”, “intend” and “plan” and similar ex­pres­sions identify for­ward looking state­ments. Actual results or per­form­ance may differ ma­teri­ally from any future results or per­form­ance ex­pressed or implied by such state­ments. The im­por­tant factors that could cause our actual results or per­form­ance to differ ma­teri­ally in­clude, among others, risks asso­ci­ated with pre-clinical and clin­i­cal devel­op­ment of prod­ucts, un­cer­tain­ties related to the out­come and conduct of clin­i­cal trials in­­clud­ing un­fore­seen safety issues, un­cer­tain­ties related to prod­uct manu­fac­tur­ing, the lack of mar­ket acceptance of our prod­ucts, our in­abil­ity to man­age growth, the competitive en­viron­ment in rela­tion­ to our business area and mar­kets, our in­abil­ity to attract and retain suitably qualified per­son­nel, the un­en­force­ability or lack of protection of our patents and pro­pri­e­tary rights, our rela­tion­ships with affiliated entities, changes and devel­op­ments in tech­nology which may render our prod­ucts obsolete, and other factors. For a further discussion of these risks, please refer to the risk man­age­ment sections in Genmab’s most recent financial reports, which are avail­able on www.genmab.com. Genmab does not under­take any obli­ga­tion to update or revise for­ward looking state­ments in this Company Announcement nor to con­firm such state­ments in rela­tion­ to actual results, unless required by law.

Genmab A/S and its sub­sid­i­aries own the fol­low­ing trademarks: Genmab®; the Y-shaped Genmab logo®; Genmab in com­bi­na­tion with the Y-shaped Genmab logo™; the DuoBody logo®; the HexaBody logo™; HuMax®; HuMax-CD20®; DuoBody®; HexaBody® and UniBody®. Arzerra® is a trademark of Novartis Pharma AG.

References

¹ American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma. Accessed August 2015.
² National Cancer Institute. "A Snapshot of Myeloma." Available at www.cancer.gov/research/progress/snapshots/myeloma. Accessed August 2015.
³ American Cancer Society. "What are the key statistics about multiple myeloma?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-key-statistics. Accessed August 2015.
⁴ GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide. Available at: http://globocan.iarc.fr/old/burden.asp?selection_pop=224900&Text-p=World&selection_cancer=17270&Text-c=Multiple+myeloma&pYear=3&type=0&window=1&submit=%C2%A0Execute. Accessed August 2015.
⁵ American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed August 2015.
⁶ Kumar, SK et al. Leukemia. 2012 Jan;26(1):149-57.
⁷ Michael de Weers et al. Dara­tu­mu­mab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. February 1, 2011vol. 186 no. 3 1840-1848.
⁸ Yulian Khagi and Tomer M Mark. Potential role of dara­tu­mu­mab in the treat­ment of multiple myeloma. Onco Targets Ther. 2014; 7: 1095–1100.
⁹ Jing Yang and Qing Yi. Therapeutic mono­clonal anti­bodies for multiple myeloma: an update and future perspectives. Am J Blood Res. 2011; 1(1): 22–33.

Source: Genmab.